Neuromuscular

The chief medical officer at Cure SMA detailed the importance of having options for patients and how certain administration routes and schedules offer more personalized approaches, particularly for adult patients with SMA.

Leah Croll, MD, a neurology resident on the front lines of the COVID-19 pandemic, shares details from her study on the psychosocial impact on physicians and her first-hand experience.

The investigator at the Healey & AMG Center for ALS at Massachusetts General Hospital spoke to the CENTAUR open-label extension and AMX0035’s development.

The Project ALS-funded oral MAP4 kinase inhibitor has been described in the literature as an exceptionally potent, metabolically stable, and blood-brain barrier-penetrant compound.

Researchers noted that the findings are encouraging for the use of real-world data and natural history data to augment, or possibly replace, placebo controls in DMD trials.

The neurology resident at NYU Langone described psychosocial implications that physicians faced during the COVID-19 pandemic and the toll it takes on them.

The investigator at the Healey & AMG Center for ALS detailed the combination of AMX0035 and other agents and how ALSFRS-R scores translate to daily life.

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending September 4, 2020.

The investigator at the Healey & AMG Center for ALS spoke to the newly released findings of the phase 2/3 trial of the investigational combination ALS agent AMX0035.

Data from the CENTAUR trial showed that the monthly rate of decline measured by ALSFR-S was 0.42 points slower for patients treated with the combination agent AMX0035.

The chief medical officer at Cure SMA detailed the importance of having various treatment options for patients, particularly for adults with SMA.

The chief medical officer at Cure SMA offers insight on the current SMA therapeutic landscape and what the organization is focused on going forward.

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending August 28, 2020.

With a positive track record in ALS, the investigational agent now sets its sights on the Alzheimer disease pipeline.

Efforts to cut through regulatory red tape will help ensure earlier access to potentially lifesaving treatments.

Neurology News Network for the week ending August 22, 2020.

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending August 21, 2020.

Qutenza becomes the first topical treatment with a TRPV1 agonist indicated for the treatment of neuropathic pain associated with diabetic peripheral neuropathy.

The co-director of the Neurological Clinical Research Institute at Massachusetts General Hospital offered her perspective on the expected developments for ALS care in next few years, and where AMX0035 might fit in that landscape.

The presence of laryngeal movement disorders—identified in 93% of the MSA group—may serve as a diagnostic biomarker of MSA independent of phenotype, disease duration, or patient age.

Neurology News Network for the week ending August 15, 2020.

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending August 14, 2020.

"Mind Moments," a podcast from NeurologyLive, brings you an exclusive interview with Richard Finkel, MD.

Advocacy organization Cure SMA provides guidance for health care providers caring for patients with SMA during the ongoing COVID-19 pandemic.

The drug is indicated in patients with a confirmed mutation in the DMD gene that is amenable to exon 53 skipping.

The co-director of the Neurological Clinical Research Institute at Massachusetts General Hospital spoke to findings of the phase 2 CENTAUR trial of AMX0035 in amyotrophic lateral sclerosis.

After receiving fast track designation by the FDA in January 2018, pegunigalsidase alfa now has a PDUFA date scheduled for January 27, 2021.

Adding to a flurry of pipeline movement since July, 2 more rare disease agents—VTA-110 for DMD and arimoclomol for NPC—have made positive steps in their clinical development recently.

The agent, marketed under the brand name Evrysdi, is the first at-home therapy indicated for treatment of adults and children with SMA.

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending August 7, 2020.